Enzyme replacement therapy (ERT) is an approved treatment strategy for those with Pompe disease. It involves providing patients with alpha-glucosidase, which helps to break down glycogen. In addition to reducing…
Danny's Dose Family Education Event: Baton Rouge, LA October 22, 2022 FOR ADULTS and FAMILIES LIVING WITH RARE DISEASE OR CHRONIC ILLNESS REQUIRING SPECIALIZED EMERGENCY CARE and/or MEDICATIONS! 10:00 –…
Online Webinar: Fighting Against the Global Issue of Substandard (SF) and Falsified Medical Products With Dr. Bahijja Raimi-Abraham October 20, 2022 Dr Bahijja Raimi-Abraham is a pharmacist, Lecturer in Pharmaceutics…
If you’re into electronic music, you need to check out DJ Funk Puppy. This ten-year-old (who goes by Richard Tyler Greene III in his day-to-day life) is a talented DJ,…
When Branden was just a young child, he was diagnosed with a rare genetic disorder called chronic granulomatous disease (CGD). Now, at 33 years old, Branden is passionate about raising…
Families caring for medically complex children face almost unfathomable decisions, often with no prior experience to guide their thinking and acting. Second only to the clinician expertise on which…
It’s no secret that receiving a rare disease diagnosis can take excess amounts of time. Various articles share that, on average, it takes anywhere from four to nine years for…
WA State Rare Chronic Illness Support Group A supportive group for WA state residents living with rare health challenges facilitated by Carrie Pope, MSW, LICSW 2nd Friday of each month,…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
For as long as he could remember, Georgie Davey had one word that he thought described himself: “clumsy.” When he was a child, Georgie would frequently trip and fall. He…
On September 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
The new nonprofit Every Cure has been created on the basis that every drug currently on the market effects multiple areas in the body. This premise has been proven…
Before you continue, make sure you've read Part 1 of our interview, where we discussed the Lord family's story and experience with rare disease, and the development of MobiMedQR. This tool, which…
If you haven't already, don't forget to go and read Part 1 of our interview. In Part 1, David Ogman and I discuss his son Jordan's TECPR2 diagnosis, the diagnostic journey,…
When asked his most important piece of advice for families within the rare disease community, Jeffrey Lord said: Do what you can control and move on from there. Every day,…
Web-Based Rare Chronic Illness Support Group A peer support group for those living with rare health challenges led by Kerry Heckman, MSW, LICSW 1st Tuesday of each month, 4-5pm PST…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
There seems to be no limits when it comes to what a parent will do for their child. And when it came down to it, David and Stacey Ogman knew…
Over the last few years, there have been slight increases in the number of acute flaccid myelitis (AFM) cases throughout the United States. Specifically, 2014, 2016, and 2018 all saw…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
Danny's Dose Family Education Event: Hot Springs, AR October 1, 2022 EDUCATION: Learn the "STEPS TO PROTECTION" for proper EMERGENCY PREPAREDNESS, "TREATMENT PLANS", working with your EMS/ER Staff, your existing…
FBXL4-related mitochondrial disease urgently requires new therapies. Unfortunately, this condition is often fatal by early ages (early to mid-childhood). Therefore, it is important to spur research into potential interventions or…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
At 20 years old, Grace Hinchman was - for all intents and purposes - a happy and healthy young adult. Grace was studying Business at Loyola University Chicago and playing…
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